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NCT02117713 · ClinicalTrials.gov registry record · Phase 2
An Extension Study to Evaluate the Long-Term Safety and Durability of Effect of LUM001 in the Treatment of Cholestatic Liver Disease in Pediatric Subjects With Alagille Syndrome
A Phase 2 study of Alagille Syndrome, sponsored by Mirum Pharmaceuticals.
- Completed
- Registry status
- Phase 2
- Development phase
- 34
- Enrollment target
- 11
- Study locations
NCT02117713: Completed Phase 2 study of Alagille Syndrome, sponsored by Mirum Pharmaceuticals.
NCT02117713 is a Phase 2 study of Alagille Syndrome that has completed, run by Mirum Pharmaceuticals. The registered enrollment target is 34 participants, below the 57-participant average among 3 other Alagille Syndrome trials with a reported enrollment target (40% lower). The trial reports 11 study locations across 9 states. According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT02117713, a Phase 2 study of Alagille Syndrome, has completed, sponsored by Mirum Pharmaceuticals.
- COMPLETED
- Registry status
- Phase 2
- Development phase
- 34 participants
- Enrollment target
- 11
- Study locations
Study Summary
This is a multicentre, extension study of LUM001 in children diagnosed with Alagille Syndrome who have completed participation in a core LUM001 treatment protocol. The primary objective is to evaluate long-term safety and tolerability of LUM001. Efficacy will be assessed by evaluating the effect of LUM001 on the biochemical markers and pruritus associated with Alagille Syndrome.
Primary Outcome
This primacy efficacy endpoint is the mean change from MRX baseline to week 48 in fasting sBA levels.
Conditions Studied
Interventions
- DRUG LUM001 (Maralixibat)
Study Locations (11)
California
- Children's Hospital Los Angeles - Los Angeles
- University of California at San Francisco Children's Hospital - San Francisco
Pennsylvania
- The Children's Hospital of Philadelphia - Philadelphia
- Children's Hospital of Pittsburgh of UPMC - Pittsburgh
Colorado
- Children's Hospital Colorado - Aurora
Indiana
- Riley Hospital for Children - Indianapolis
Ohio
- Cincinnati Children's Hospital Medical Center - Cincinnati
Texas
- Baylor College of Medicine/Texas Children's Hospital - Houston
Utah
- University of Utah - Salt Lake City
Washington
- Seattle Children's Hospital - Seattle
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 34 participants |
| Start Date | 2015-03-16 |
| Est. Completion | 2020-06-01 |
| Phase | Phase 2 |
What the finished NCT02117713 record still lists
NCT02117713 is an interventional study that assigns participants to a tested intervention. The registry caps enrollment at 34 participants, a relatively small participant target, below the 57-participant average among 3 other Alagille Syndrome trials with a reported enrollment target (40% lower).
The record links to 1 condition, with Alagille Syndrome appearing as the primary indexed condition, and to 1 intervention - of which LUM001 (Maralixibat) is the first listed.
NCT02117713 lists 11 locations in 9 states (California, Pennsylvania, Colorado).
Frequently Asked Questions
What is clinical trial NCT02117713 about?
NCT02117713 is a clinical study titled "An Extension Study to Evaluate the Long-Term Safety and Durability of Effect of LUM001 in the Treatment of Cholestatic Liver Disease in Pediatric Subjects With Alagille Syndrome". This is a multicentre, extension study of LUM001 in children diagnosed with Alagille Syndrome who have completed participation in a core LUM001 treatment protocol. The primary objective is to evaluate long-term safety and tolerability of LUM001. Efficacy will be assessed by evaluating the effect of ...
What is the current status of trial NCT02117713?
This trial is currently completed. It is a Phase 2 study. The enrollment target is 34 participants. The study started on 2015-03-16. Estimated completion is 2020-06-01.
What conditions does trial NCT02117713 study?
This clinical trial studies the following conditions: Alagille Syndrome.
What interventions are being tested in trial NCT02117713?
The interventions under investigation include: LUM001 (Maralixibat) (DRUG).
Who is sponsoring clinical trial NCT02117713?
This trial is sponsored by Mirum Pharmaceuticals, which has 28 total clinical trials registered on ClinicalTrials.gov.
Where is trial NCT02117713 being conducted?
This trial has 11 study locations across California, Colorado, Indiana, Ohio, Pennsylvania. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.
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